Helping Beethoven hear

Little progress has been made in developing therapies for hearing loss that go beyond the temporary and partial solutions provided by hearing aids and cochlear implants, and most company activity is focused on acquired forms of the disease. Now, a group at Boston Children's Hospital has looked at genetic causes of deafness and designed a gene therapy to restore function in TMC1, one of the disease's most commonly mutated targets.

Attempting gene therapy is still relatively uncommon in hearing loss. Novartis AG and GenVec Inc. have the CGF166 gene therapy in Phase I/II testing for acquired hearing loss, but the majority of companies in the space have programs based on small molecules. (See Table: Commercial programs in hearing loss)...